Parents of children with arthritis know the dilemma all too well. While biologic response modifiers offer unparalleled benefits in the short term, they have the potential for unsettling long-term side effects. At least we think so – although biologics have been approved for use with kids since 1999, there’s no national database that tracks patients’ progress with the disease and their response to various medications.

That changed in the summer of 2010, which saw the debut of a database specifically designed to gather such details. As the project’s principal investigator, Laura Schanberg, MD, co-chief of Duke University’s division of pediatric rheumatology, has overseen infrastructure development and rollout via institutions that are part of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), a national network of pediatric rheumatology researchers.

Dr. Schanberg reports that CARRA-net, as the database is named, is up and running at six sites and adding more patients every week toward its initial goal of 20,000. At present, participation is open to kids with JIA, lupus, vasculitis, dermatomyositis, fibromyalgia, systemic scleroderma, localized scleroderma and sarcoidosis who are being treated at a CARRA-affiliated facility (most are university health care centers or pediatric health care facilities). Enrollment consists of filling out consent forms and questionnaires, and takes about 15 minutes, Dr. Schanberg says. Patient health information is entered into the database and updated every six months. The plan is to make participation available at all 60 CARRA sites nationwide, then to all pediatric rheumatologists nationwide, and then to non-specialists who, due to location wind up treating kids with rheumatic conditions.

That’s important because kids in many states may not have access to a pediatric rheumatologist, says Bernard Murphy, chair of the Arthritis Foundation’s Juvenile Arthritis Leadership Group, which directs the Juvenile Arthritis Alliance. “They’re likely to be treated by an adult rheumatologist or even a general practitioner – someone who may not be up to speed on the JA disease model and treatment options.”

As the leader of “open-mic” nights at JIA advocacy summits, during which parents are encouraged to share their concerns about biologics, specifically those known as tumor necrosis factor (TNF) inhibitors, Murphy says he’s come across one question time and again: Will this treatment shorten my child’s life expectancy or put them at risk of developing cancer?

CARRA-net is the first step toward answering that difficult question. “You get that diagnosis, and there will be a system in place that says ‘This is the best course of treatment that we know of based on the fact that we’ve been looking at kids over time who are on different therapies,’” says Amy Melnick, the Arthritis Foundation’s vice president of public policy. “There’s so much more potential to improve care when you can aggregate this data.”

Expanding the reach

The idea isn’t new, Melnick says. The FDA convened a public workshop that started the ball rolling in the spring of 2009.

Initial funding for CARRA-net came last year in the form of $7 million in stimulus funds from the National Institutes of Health. Prior to its development, pharmaceutical companies monitored patients who were taking their medications, but that tracking stopped once a patient changed therapies – something that happens quite often with arthritis patients of all ages.

Juvenile Idiopathic Arthritis Registry Launched

Parents of children with arthritis know the dilemma all too well. While biologic response modifiers offer unparalleled benefits in the short term, they have the potential for unsettling long-term side effects. At least we think so – although biologics have been approved for use with kids since 1999, there’s no national database that tracks patients’ progress with the disease and their response to various medications.

That changed in the summer of 2010, which saw the debut of a database specifically designed to gather such details. As the project’s principal investigator, Laura Schanberg, MD, co-chief of Duke University’s division of pediatric rheumatology, has overseen infrastructure development and rollout via institutions that are part of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), a national network of pediatric rheumatology researchers.

Dr. Schanberg reports that CARRA-net, as the database is named, is up and running at six sites and adding more patients every week toward its initial goal of 20,000. At present, participation is open to kids with JIA, lupus, vasculitis, dermatomyositis, fibromyalgia, systemic scleroderma, localized scleroderma and sarcoidosis who are being treated at a CARRA-affiliated facility (most are university health care centers or pediatric health care facilities). Enrollment consists of filling out consent forms and questionnaires, and takes about 15 minutes, Dr. Schanberg says. Patient health information is entered into the database and updated every six months. The plan is to make participation available at all 60 CARRA sites nationwide, then to all pediatric rheumatologists nationwide, and then to non-specialists who, due to location wind up treating kids with rheumatic conditions.

That’s important because kids in many states may not have access to a pediatric rheumatologist, says Bernard Murphy, chair of the Arthritis Foundation’s Juvenile Arthritis Leadership Group, which directs the Juvenile Arthritis Alliance. “They’re likely to be treated by an adult rheumatologist or even a general practitioner – someone who may not be up to speed on the JA disease model and treatment options.”

As the leader of “open-mic” nights at JIA advocacy summits, during which parents are encouraged to share their concerns about biologics, specifically those known as tumor necrosis factor (TNF) inhibitors, Murphy says he’s come across one question time and again: Will this treatment shorten my child’s life expectancy or put them at risk of developing cancer?

CARRA-net is the first step toward answering that difficult question. “You get that diagnosis, and there will be a system in place that says ‘This is the best course of treatment that we know of based on the fact that we’ve been looking at kids over time who are on different therapies,’” says Amy Melnick, the Arthritis Foundation’s vice president of public policy. “There’s so much more potential to improve care when you can aggregate this data.”

Expanding the reach

The idea isn’t new, Melnick says. The FDA convened a public workshop that started the ball rolling in the spring of 2009.

Initial funding for CARRA-net came last year in the form of $7 million in stimulus funds from the National Institutes of Health. Prior to its development, pharmaceutical companies monitored patients who were taking their medications, but that tracking stopped once a patient changed therapies – something that happens quite often with arthritis patients of all ages.

When the stimulus funds run out in the fall of 2011, the project’s leaders hope to have another funding mechanism in place – the drug companies themselves. What’s the incentive for getting these manufacturers – who may be leery of research that indicates one medication is better than another – to participate? That’s where the FDA comes in.

“What they can do is mandate that the manufacturers participate in and help fund this registry,” Melnick says. It’s a simple equation – you want FDA approval for your next arthritis medication? Help fund the registry.

Murphy also is seeking additional funding outside the medical and pharmaceutical arena. “We’re trying to broaden our reach outside pharma into other groups that may not realize they’re stakeholders,” he says. “Arthritis-related loss of productivity can affect almost any industry.”

While there is no precedent for such a registry in the arthritis community, there is Children’s Oncology Group (COG) which grew out of clinical trials and now encompasses 200 cancer treatment institutions worldwide. In 2001 the National Cancer Institute funded COG’s registry effort, the Childhood Cancer Research Network (CCRN), which functions much the same as CARRA-net.

At present, the potential database for JIA patients hovers around 300,000 in the United States. However, Murphy says European scientists are working with those in the U.S. to integrate databases, thus upping the sample size. “If you can integrate it worldwide, or at least with the E.U., Canada and perhaps Latin America, the sample size becomes larger and much better.”

Murphy, whose daughter Kate, now 18, was diagnosed with JIA at age 6, takes a long-term view of the effort. “I recognize at this point – as do many of the folks who have been in this for a while – that this isn’t going to help my child. But over 10 years or 15 years, the next few generations of kids coming up with this disease are going to be helped tremendously. That really gets to the heart of it with parents. There’s a sense of relief in the room when you can say that your government is behind this and there’s a big group of very dedicated individuals working toward getting this done.”

What is the CARRA Registry?

The CARRA Registry is a database of patients with pediatric rheumatic diseases. Researchers will study information in the database to improve future treatments and health outcomes and maybe even prevent or cure pediatric rheumatic diseases in the future. The registry will also help identify patients who may be eligible for future studies. The CARRA Registry plans to enroll about 20,000 patients at about 60 CARRA centers.

Who is eligible?

Your child may be eligible if he/she is 21 years old or younger and was diagnosed with a rheumatic disease before the age of 16.

What will happen at CARRA Registry visits?

At your child’s first visit you will be asked to:

• Review and sign a consent form

• Answer questions about your child’s medical and family history and how his/her medical condition affects his/her life

• Provide contact information for yourself and other persons who know how to contact you and your child

At follow-up visits (approximately two visits per year) you will be asked to: